Regen BioPharma Inc. (OTCQB: RGBP), (OTCQB: RGBPP) President and Chief Scientific Officer Harry Lander provides clarity for stakeholders about the therapeutic drug development process. Therapeutic drugs come in many forms, such as small molecules, biologics, gene therapies, nucleic acids, cell therapies and vaccines. Of these, only small molecule drugs have the most well-defined process for getting to market and possess the advantages inherent in the ability to control the chemistry involved in the manufacture of the drug.

The typical pathway for creating a small molecule drug involves several steps and can take many years. In fact, taking a drug from an initial idea to market-ready takes, on average, 15 years and well over $1 billion.

However, once a lead is identified, the timeline to submission to the FDA for permission to test the drug in humans can be as little as two years. Lander explains each of these steps below so the complexity, expense and time commitment can be better understood by the company's shareholders and other stakeholders.

Target Validation:

This is typically the most time-consuming part of the process and can take many years. During this step, the target is proven to be critical for the disease being studied and its susceptibility to attack by drugs is evaluated. The experiments utilized in this phase include cellular experiments and animal studies. In the case of NR2F6, Regen scientists and other academic groups unrelated to Regen have published papers going back to 2011 identifying NR2F6 as a critical factor in immune and cancer stem cell regulation.

High Throughput Screening:

The fastest way to identify small molecules that can bind to and alter the function of a target is to use a process called high throughput screening (HTS). By using this process, it is possible to quickly screen hundreds of thousands of potential small molecules to see if any bind to the target, which is termed "hits". Depending on the target, it can be easy or difficult to find hits. The NR2F6 target is considered a very difficult target, and, as far as the company knows, Regen is the only organization in the world that has identified validated hits against this target. Regen began the process of HTS in early 2016.

Hit Prioritization:

Once a series of hits have been produced by screenings, it is important to analyze the data to make sure the compounds identified for further development are likely to be selective towards the target.

For example, in many cases some hits are identified which turn out to be compounds that would bind to many different targets. These compounds are not useful as a potential drug because they bind to many targets in the body. This causes many unwanted effects. So, choosing a selective hit, a compound focused on NR2F6, is key in this step. With the help of our Scientific Advisory Board and outside commercial experts in medicinal chemistry, we identified a handful of hits that we selected to advance forward for further development.

Lead Identification:

This phase is essentially a series of tests that validate the fact that the hit that has been selected is a useful starting point. One way to do this is to sequentially chemically modify the original hit compound and test it in the HTS assay. By doing this, one can see a relationship between the structure of the compound and its activity in the assay. Our HTS screening assay had several structurally-related compounds in it related to our primary hit, and these showed activity as well, suggesting that our hit was indeed an excellent starting point.

Lead Optimization:

In this phase, the goal is to make the compound stronger, better and faster. These rounds of optimization experiments are meant to increase the solubility and affinity of the compound to the target and to minimize any potential toxicities. Again, this is an iterative process and normally takes six to nine months. We are about to embark on this stage.

ADMET:

ADMET stands for absorption, distribution, metabolism, excretion and toxicology. In other words, using animal models one measures how quickly the drug is absorbed into the blood stream, how long it stays in the blood stream, where in the body it goes, how it is broken down and excreted and at what doses it is toxic. Most of this risk is mitigated in the prior phase of Lead Optimization, but here the compounds are put into animal models and directly tested.

Preclinical:

Here, the drug is tested for effectiveness in various animal models.

There are multiple places the project can fail during this process. The most common include (i) the inability to configure a reliable assay for HTS; (ii) no developable hits obtained from the HTS; (iii) compounds do not behave as desired in secondary or native tissue assays; (iv) compounds are toxic in vitro or in vivo; (v) compounds have undesirable side effects which cannot be easily screened out or separated from the mode of action of the target; and (vi) inability to obtain a good ADMET profile. Regen's program on NR2F6 has already cleared hurdles i-iii, although more testing needs to be done.

In summary, creating a small molecule drug follows a well-worn path in the pharmaceutical industry. Regen's program, although early, remains robust and is on track to complete the Lead Optimizations stage in third or fourth quarter of 2017.

About Regen BioPharma Inc.:

Regen BioPharma Inc. is a publicly traded biotechnology company (OTCQB: RGBP) and (OTCQB: RGBPP). The Company seeks to identify undervalued regenerative medicine applications in the immunotherapy and stem cell space. The Company is focused on rapidly advancing these technologies through pre-clinical and Phase I/ II clinical trials. Currently, the Company is focused on gene silencing therapy and small molecule therapies for treating cancer, along with developing stem cell treatments for aplastic anemia and disorders of the bone marrow. Additional information on Regen BioPharma is available at http://www.regenbiopharmainc.com.

Disclaimer: This news announcement may contain forward-looking statements. Forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified. Future events and actual results could differ materially from those set forth in, contemplated by, or underlying the forward-looking statements. The risks and uncertainties to which forward looking statements are subject include, but are not limited to, the effect of government regulation, competition and other material risks.